AstraZeneca: positive trial for Koselugo in neurofibromatosis
(CercleFinance.com) - AstraZeneca reports that the largest multicenter, randomized, placebo-controlled Phase III trial in adult patients with symptomatic and inoperable neurofibromatosis type 1 (NF1) with plexiform neurofibromas, showed that Koselugo (selumetinib) met its primary objective by demonstrating a significant and clinically relevant objective response rate (ORR) compared with placebo.
NF1, a rare genetic disease affecting around 1.7 million people worldwide, often leads to debilitating symptoms.
Koselugo, the only targeted treatment approved for children with NF1, could now benefit adults with no other therapeutic option.
Alexion (AstraZeneca's rare disease company) and MSD will present these data to regulatory authorities for possible extension of its use.
Copyright (c) 2024 CercleFinance.com. All rights reserved.
NF1, a rare genetic disease affecting around 1.7 million people worldwide, often leads to debilitating symptoms.
Koselugo, the only targeted treatment approved for children with NF1, could now benefit adults with no other therapeutic option.
Alexion (AstraZeneca's rare disease company) and MSD will present these data to regulatory authorities for possible extension of its use.
Copyright (c) 2024 CercleFinance.com. All rights reserved.